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New treatment option for Severe Aplastic Anemia
Official title Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
ClinicalTrials.gov ID: NCT01174108
What this study is testing
- What it's testing
- Background: Stem cell transplants from related donors (allogenic stem cell transplants) can be used to treat individuals with certain kinds of severe blood diseases or cancers, such as severe anemia. Allogenic stem cell transplants encourage the growth of new bone marrow to replace that of the recipient.
- Phase 2: a mid-size study of how well it works
- Time commitment: about 3 years
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 80
You may be able to join if
- Recipient:
- Patients diagnosed with one of the following hematologic diseases which are associated with reasonable longevity, shown to be curable by allogeneic...
- 1) Paroxysmal nocturnal hemoglobinuria (PNH) associated with life-threatening thrombosis, and/or cytopenia, and/or transfusion dependence and/or...
- 2) Severe aplastic anemia (SAA) or pure red cell aplasia (PRCA [acquired or congenital]) with bone marrow cellularity \<30% (excluding lymphocytes)...
- 3) Refractory anemia (RA) or RARS MDS patients who have associated transfusion dependence and/or neutropenia.
You likely can't join if
- \- Recipient: any of the following
- Major anticipated illness or organ failure incompatible with survival from PBSC transplant
- Diffusion capacity of carbon monoxide (DLCO) \<40% predicted (patients under the age of 10 may be excluded from this criterion if they have...
- Left ventricular ejection fraction \<40% (evaluated by ECHO)
- Serum creatinine greater than 2.5mg/dl or creatinine clearance less than 50 ml/min by 24 hr urine collection
- Serum bilirubin greater than 4 mg/dl, transaminases greater than 5 times the upper limit of normal
See the full eligibility criteria
- Recipient:
- Patients diagnosed with one of the following hematologic diseases which are associated with reasonable longevity, shown to be curable by allogeneic BMT but where concern for a high procedural mortality with conventional...
- 1) Paroxysmal nocturnal hemoglobinuria (PNH) associated with life-threatening thrombosis, and/or cytopenia, and/or transfusion dependence and/or recurrent and debilitating hemolytic crisis
- 2) Severe aplastic anemia (SAA) or pure red cell aplasia (PRCA [acquired or congenital]) with bone marrow cellularity \<30% (excluding lymphocytes) associated with RBC or platelet transfusion dependence and/or...
- 3) Refractory anemia (RA) or RARS MDS patients who have associated transfusion dependence and/or neutropenia.
- Ages 4 to 80 (both inclusive), and weight \>15 kg
- Availability of HLA identical or single HLA locus mismatched family donor or 10/10 matched unrelated donor at the allelic level (HLA alleles A, B, C, DR, and DQ).
- 9/10 donors where all the HLA sequences have the same antigen/peptide binding domains in key exons to the patient. This can result in identical protein sequences between patient and donor. Allele mismatches in p and g...
- Telomere Length Testing
- Germline/Inherited gene panel in patients where a suspicion for a familial bone marrow failure syndrome (BMFS) exist, hTERC and hTERT, GATA2 mutation testing will be performed on protocol 04-H-0012 or performed...
- \- Recipient: any of the following
- Major anticipated illness or organ failure incompatible with survival from PBSC transplant
- Diffusion capacity of carbon monoxide (DLCO) \<40% predicted (patients under the age of 10 may be excluded from this criterion if they have difficulty performing the test correctly and thus are unable to have their DLCO...
- Left ventricular ejection fraction \<40% (evaluated by ECHO)
- Serum creatinine greater than 2.5mg/dl or creatinine clearance less than 50 ml/min by 24 hr urine collection
- Serum bilirubin greater than 4 mg/dl, transaminases greater than 5 times the upper limit of normal
- Pregnant or lactating
- Fanconi s anemia (test to be performed at a CLIA-certified laboratory)
- ECOG performance status of 3 or more (See NIH Bone \& Marrow Transplant Consortium Supportive Care Guidelines for HSCT Recipients or Institutional Guidelines for bone and marrow transplants)
- Other malignant diseases liable to relapse or progress within 5 years, with the exception of a separate hematologic malignancy where allogeneic stem cell transplant has been shown to be potentially curative.
- Presence of an active infection not adequately responding to appropriate therapy.
- Inability to comprehend the investigational nature of the study and provide informed consent. The procedure will be explained to people age 8 -17 years with formal consent being obtained from parents or legal guardian...
- Related donor deemed suitable and eligible, and willing to donate, per clinical evaluations who are additionally willing to donate blood samples for research. Related donors will be evaluated in accordance with existing...
- Age greater than or equal to 4 and less than or equal to 80 years old EXCLUSION CRITERIA: -Related Donor: None INCLUSION CRITERIA \& EXCLUSION CRITERIA: Unrelated Donor \- The NMDP unrelated donor inclusion criteria...
The study team makes the final eligibility decision.
Where it's taking place
- Baltimore, Maryland, United States
- Bethesda, Maryland, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 3 years per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 4 years to 80 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Baltimore, Maryland, United States; Bethesda, Maryland, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.